A comprehensive care pathway of gene therapy for hemophilia based on current guideline documents and summary of product characteristics: communication from the ISTH SSC working group on gene therapy
Based on SMPCs and (inter)national guidance documents and recommendations a care pathway has been developed and visualized in a Metro Map. This provides a clear and comprehensive overview of all activities, contact moments, and responsibilities within the longitudinal gene therapy treatment process. This comprehensive care pathway may help navigate gene therapy implementation, providing guidance to clinicians, patients, and caregivers.
Authors
- Frank W.G. Leebeek (ORCID: https://orcid.org/0000-0001-5677-1371)
- Alok Srivastava (ORCID: https://orcid.org/0000-0001-5032-5020)
- Radosław Kaczmarek (ORCID: https://orcid.org/0000-0001-8084-1958)
- Flora Peyvandi (ORCID: https://orcid.org/0000-0001-7423-9864)
- David Lillicrap (ORCID: https://orcid.org/0000-0003-2410-6312)
- Pratima Chowdary (ORCID: https://orcid.org/0000-0002-6690-8586)
- Ilaria Cutica (ORCID: https://orcid.org/0000-0003-2749-0719)
- Jan Voorberg (ORCID: https://orcid.org/0000-0003-4585-2621)
- Glenn F. Pierce (ORCID: https://orcid.org/0000-0002-3310-328X)
- Wolfgang Miesbach (ORCID: https://orcid.org/0000-0002-4506-0061)
- Johnny Ndoni Mahlangu (ORCID: https://orcid.org/0000-0001-5781-7669)
- Caroline Mussert (ORCID: https://orcid.org/0009-0009-8970-9473)
- Paul Batty (ORCID: https://orcid.org/0000-0002-7808-3462)
- Amit Nathwani
- Steven W Pipe
Institutions
- Twitter (United States) (US)
- Goethe University Frankfurt (DE)
- University of Milan (IT)
- University of the Witwatersrand (ZA)
- Queen's University (CA)
- University of Michigan (US)
- The Royal Free Hospital (GB)
- Canadian Hemophilia Society (CA)
- Erasmus MC (NL)
- Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico (IT)
- Sanquin (NL)
- University Hospital Frankfurt (DE)
- Erasmus MC - Sophia Children’s Hospital (NL)
- St.John's Medical College Hospital (IN)
- Ludwik Hirszfeld Institute of Immunology and Experimental Therapy (PL)
- University College London (GB)
- Indiana University – Purdue University Indianapolis (US)
- Erasmus University Rotterdam (NL)
Publication Details
- Journal
- Journal of Thrombosis and Haemostasis
- Published
- 2025-10-27
- DOI
- https://doi.org/10.1016/j.jtha.2025.09.041
- Primary Topic
- Hemophilia Treatment and Research
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- Nederlandse Organisatie voor Wetenschappelijk Onderzoek